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WBUR reports on early results for gene therapy trial for two young children with Tay-Sachs disease

Treatment was developed at UMass Medical School and licensed to Axovant Gene Therapies

Terence Flotte
Terence R. Flotte, MD

Two young children with Tay-Sachs disease were treated safely at UMass Memorial Medical Center with a gene therapy developed at UMass Medical School and, in one case, the child’s condition has stabilized, according to an interview published by WBUR with Terence R. Flotte, MD.

Dr. Flotte, the Celia and Isaac Haidak Professor of Medical Education, executive deputy chancellor, provost and dean of the School of Medicine, presented the data from an expanded access study of the investigational gene therapy on Wednesday, Oct. 23, in Barcelona at the European Society of Gene and Cell Therapy Annual Congress.

“It seems right now that she’s not degenerating,” Flotte told WBUR, of the second patient who was only about six months old when recently treated. “But I would say it’s too early to say that definitively.”

He said the first patient, who had advanced disease when treated at age two and a half, has seen a biochemical effect but no clinical improvement.

Tay-Sachs is a rapidly progressive and fatal pediatric neurodegenerative genetic disorder that has a median life expectancy of approximately three to four years. In 2018, Axovant Gene Therapies licensed exclusive worldwide rights from UMass Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases. Research into potential therapies for lysosomal storage diseases such as Tay-Sachs, Sandhoff disease and GM1 gangliosidosis at UMass Medical School and Auburn University has led to significant advances in the field. Flotte said Axovant is planning a Phase 2 trial.

Read the full WBUR story here:
Early Report: Baby Treated With Gene Therapy For Deadly Tay-Sachs Disease Appears To Stabilize

Related stories on UMassMedNow:
First evidence of clinical stabilization in Tay-Sachs presented at European Society of Gene & Cell Therapy Congress
LISTEN: New hope for Tay-Sachs disease at UMass Medical School
UMMS licenses clinical-stage gene therapies for Tay-Sachs, similar diseases to Axovant